News & Stories
See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.
Dr. Charles Abrams Discovers That Inosine Treatment Leads to Benefits in a CMT1X Animal Model
Dr. Charles Abrams, Professor of Neurology and Rehabilitation at the University of Illinois, Chicago, has discovered that inosine could potentially reduce nerve inflammation and offer moderate functional benefits in preclinical animal models of Charcot-Marie-Tooth...
CMT Research Foundation Invests in XtRNA Bio to Develop Gene Therapy for CMT2D
CMT Research Foundation has invested in a research project with XtRNA Bio aimed to develop a new viral gene therapy for Charcot-Marie-Tooth disease type 2D. How GARS1 Mutations Drive CMT2D CMT2D is caused by mutations in the GARS1 gene, which encodes glycyl-tRNA...
CMT Research Foundation Invests in Asha Therapeutics to Test Novel Drug for CMT2A
CMT Research Foundation has invested in a research project with Asha Therapeutics to investigate the therapeutic potential of inhibiting SARM1 (Sterile alpha and TIR motif-containing protein 1) using their novel drug, ASHA-624, for the treatment of Charcot-Marie-Tooth...
Vanderbilt University Researchers Identify Promising Compounds Targeting PMP22 Protein Expression, Paving the Way for New Treatments for CMT1A, CMT1E and HNPP
Researchers at Vanderbilt University have made significant progress in the development of small molecules aimed at treating Charcot-Marie-Tooth disease types caused by mutations in the PMP22 gene, which include CMT1A, CMT1E and hereditary neuropathy with liability to...
Dr. Alessandra Bolino Unravels Complexities and Tests Treatment Strategies in CMT4B1
CMT4B1 is a severe form of Charcot-Marie-Tooth disease caused by mutations in the gene that makes Myotubularin-related protein 2 (MTMR2). Dr. Alessandra Bolino, PhD, discovered that mutations in this gene affect the production of fats, called phospholipids. Among...
82VS Progressing Ahead of Schedule in CMTRF-Funded Project to Improve Oligonucleotide Drug Delivery for CMT
Since launching their collaboration with CMT Research Foundation in November 2024, researchers at 82VS, the venture studio of Alloy Therapeutics, have made rapid progress and are ahead of schedule testing their novel technology focused on improving RNA therapeutic...
Initial CMTRF Funding Leads Augustine Therapeutics to Raise $85 Million for CMT Clinical Trials
CMT Research Foundation funded partner Augustine Therapeutics recently announced that they completed their Series A financing round and raised $85 million, which will be used to advance their Charcot-Marie-Tooth disease drug candidate, AGT-100216, through a Phase I/II...
CMT Research Foundation Funds Project with Alloy Therapeutics’ 82VS to Test Novel RNA Therapeutics for Charcot-Marie-Tooth Disease
The CMT Research Foundation has invested over $500,000 in a program led by 82VS, the venture studio of Alloy Therapeutics, Inc. to discover and develop novel Antibody Oligonucleotide Conjugate (AOC) therapeutics for CMT. Oligonucleotide therapeutics offer promise for...
Dr. Alessandra Bolino Completes Project Testing PIKfyve Inhibitor Drug from AcuraStem in CMT4B1 models
Previous research from Dr. Alessandra Bolino demonstrated that inhibiting a protein called PIKfyve kinase could ameliorate some disease characteristics in CMT4B1 cells. In this project, Dr. Bolino partnered with AcuraStem to test one of their PIKfyve inhibitor drug...
CMT Research Foundation Invests in Project at Nationwide Children’s Hospital to Develop Improved Gene Therapy Delivery Systems for Several Types of CMT
The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has invested in a research project to develop new vehicles for delivering gene therapies more efficiently into peripheral nerves. This...